Tags » Hemophilia A

Back in the hospital

It’s been a while since I’ve blogged. My poor hemophiliac son is back in the hospital and it’s for an infection. We’ve already been here for one week.


Rare Disease Start-Up: Dimension Therapeutics’ Gene Therapy For Hemophilia A

This Blog Post reviews the new start-up, Dimension Therapeutics and the company’s first gene therapy program for Hemophilia A.

The creation of Dimension Therapeutics (DT), a new Cambridge MA-based start-up gene therapy company, focusing on the development of treatments for rare diseases, is announced in… 411 more words

Rare Diseases

Orphan Drugs: FDA Approval of Baxter’s Treatment For Rare Form of Hemophilia

On October 24th, the FDA announces the approval of Baxter International’s orphan drug Obizur for the treatment of bleeding episodes in adults with Acquired Hemophilia A (AHA) (acquired Factor VIII deficiency). 266 more words

Orphan Drug Development